MX2022016332A - Administracion de vectores de virus adenoasociados para distrofias musculares. - Google Patents

Administracion de vectores de virus adenoasociados para distrofias musculares.

Info

Publication number
MX2022016332A
MX2022016332A MX2022016332A MX2022016332A MX2022016332A MX 2022016332 A MX2022016332 A MX 2022016332A MX 2022016332 A MX2022016332 A MX 2022016332A MX 2022016332 A MX2022016332 A MX 2022016332A MX 2022016332 A MX2022016332 A MX 2022016332A
Authority
MX
Mexico
Prior art keywords
adeno
associated virus
virus vector
muscular dystrophies
vector delivery
Prior art date
Application number
MX2022016332A
Other languages
English (en)
Inventor
Rachael Potter
Jerry R Mendell
Louise Rodino-Klapac
Danielle Griffin
Ellyn Peterson
Original Assignee
Res Inst Nationwide Childrens Hospital
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of MX2022016332A publication Critical patent/MX2022016332A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2887—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD20
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Ultra Sonic Daignosis Equipment (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La descripción proporciona un método para tratar la distrofia muscular, en un sujeto que lo necesita, que comprende la administración de un vector de terapia génica, como un vector de virus adenoasociado (AAV), que expresa un gen de microdistrofina humana miniaturizada en combinación con una etapa de supresión del sistema inmunitario del sujeto.
MX2022016332A 2020-06-15 2021-06-15 Administracion de vectores de virus adenoasociados para distrofias musculares. MX2022016332A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063039252P 2020-06-15 2020-06-15
US202063083953P 2020-09-27 2020-09-27
US202163160376P 2021-03-12 2021-03-12
US202163188266P 2021-05-13 2021-05-13
PCT/US2021/037470 WO2021257595A1 (en) 2020-06-15 2021-06-15 Adeno-associated virus vector delivery for muscular dystrophies

Publications (1)

Publication Number Publication Date
MX2022016332A true MX2022016332A (es) 2023-04-03

Family

ID=77051123

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022016332A MX2022016332A (es) 2020-06-15 2021-06-15 Administracion de vectores de virus adenoasociados para distrofias musculares.

Country Status (13)

Country Link
US (1) US20210393801A1 (es)
EP (1) EP4164668A1 (es)
JP (2) JP2023530974A (es)
KR (1) KR20230035043A (es)
CN (1) CN116348149A (es)
AU (1) AU2021293197A1 (es)
BR (1) BR112022025586A2 (es)
CA (1) CA3187353A1 (es)
CO (1) CO2023000156A2 (es)
IL (1) IL299094A (es)
MX (1) MX2022016332A (es)
TW (1) TW202208630A (es)
WO (1) WO2021257595A1 (es)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11617783B2 (en) 2015-11-16 2023-04-04 Research Institute At Nationwide Children's Hospital Repairing a mutant human titin gene using CRISPR technology
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AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
US12391928B2 (en) 2018-06-29 2025-08-19 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
CN113766935A (zh) 2019-02-26 2021-12-07 全国儿童医院研究所 β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
SI4017871T1 (sl) 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CN119630691A (zh) * 2022-06-30 2025-03-14 萨勒普塔医疗公司 治疗肌营养不良的方法
WO2024138158A2 (en) 2022-12-23 2024-06-27 Ultragenyx Pharmaceutical Inc. Method of producing soluble microdystrophin protein and uses of the same
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WO2025225719A1 (en) * 2024-04-26 2025-10-30 Chugai Seiyaku Kabushiki Kaisha Treatment and prevention of muscular dystrophy and its related diseases or disorders with satralizumab

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Publication number Publication date
JP2023530974A (ja) 2023-07-20
CA3187353A1 (en) 2021-12-23
BR112022025586A2 (pt) 2023-03-07
EP4164668A1 (en) 2023-04-19
CO2023000156A2 (es) 2023-03-27
US20210393801A1 (en) 2021-12-23
AU2021293197A1 (en) 2023-02-09
JP2026022658A (ja) 2026-02-12
TW202208630A (zh) 2022-03-01
CN116348149A (zh) 2023-06-27
WO2021257595A1 (en) 2021-12-23
IL299094A (en) 2023-02-01
KR20230035043A (ko) 2023-03-10

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